Ms. Hester is Content Specialist with Contemporary OB/GYN and Contemporary Pediatrics.
The US Food and Drug Administration (FDA) has approved the first treatment for the rare genetic disorder Molybdenum Cofactor Deficiency Type A.
The US Food and Drug Administration (FDA) has approved Nulibry (fosdenopterin) from Origin Biosciences, Inc. for the treatment of Molybdenum Cofactor Deficiency Type A, which is a rare genetic disorder that prevents a child from producing cyclic pyranopterin monophosphate.1 The disorder often presents in the first days of a child’s life and causes intractable seizures, brain injury, and subsequent death from infections in early childhood. Nulibry is the first treatment for the condition.
Nulibry is administered intravenously and replaces the missing cyclic pyranopterin monophosphate. The efficacy of the drug was shown in a study of 31 patients: 13 were treated with Nulibry and 18 received normal care. The study found that that the patients treated with Nulibry had a surivival rate of 84% at age 3 years whereas the untreated patients had a survival rate of 55% at age 3 years. The most common side effects in the study included fever, vomiting, gastroenteritis, diarrhea, respiratory infections, and complications linked to the intravenous line. Animal studies indicated potential phototoxicity, so it’s recommended that patients treated with Nulibry avoid exposure to the sun and when exposure can’t be avoided, it’s recommended that the child wear sunscreen, protective clothing, and sunglasses.
In the press release for the approval, Hylton V. Joffe, MD, MMSc, director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine in the FDA’s Center for Drug Evaluation and Research said, “Today’s action marks the first FDA approval for a therapy to treat this devastating disease. The FDA remains committed to facilitating the development and approval of safe and effective therapies for patients affected by rare diseases—an area of critical need.”
1. US Food and Drug Administration. FDA approves first treatment for molybdenum cofactor deficiency type a. Published February 26, 2021. Accessed March 1, 2021. https://www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-molybdenum-cofactor-deficiency-type